Safety, Tolerability, and Efficacy of Deferasirox in MDS

Summary

Open label, single arm study on Deferasirox treatment in MDS patients with chronic
transfusional hemosiderosis.

Patients receive daily oral dosis of Deferasirox in order to eliminate the quantity of iron
administered during transfusions and, if needed, to reduce the overload of already present
iron.

After an screening phase in which patients are evaluated according to eligibility criteria,
a one year treatment phase foresees monthly visits to evaluate safety and efficacy signs.

Description

It has been widely shown that an appropriate chelating therapy in chronic anemias
transfusion dependent can prevent the overstock of iron and can reduce the already existing
overstock reducing, then, the co-morbidity and improving survival.

In particular, some authors have shown in MDS affected patients undergoing intensive
chelating therapy with deferoxamine haematological recovery with a reduction of the need of
transfusions.

With the present study, we plan to evaluate the safety and efficacy of a therapy with the
new oral chelating Deferasirox in MDS patients with transfusional hemosiderosis.

This is an open label, single arm study on Deferasirox treatment in MDS patients with
chronic transfusional hemosiderosis.

Patients will receive daily oral dosis of Deferasirox in order to eliminate the quantity of
iron administered during transfusions and, if needed, to reduce the overload of already
present iron.

After an screening phase in which patients are evaluated according to eligibility criteria,
a one year treatment phase foresees monthly visits to evaluate safety and efficacy signs.

A d4 -labeled isotopomer of deferasirox was synthesized as internal standard for use in a LC/mass spectroscopy (MS)/MS method developed for the simultaneous quantitative determination of deferasirox i...

Recent genomic sequencing efforts have identified a number of recurrent mutations in myelodysplastic syndromes (MDS) that may contribute to disease progression and overall survival, including mutation...

Although numerous recent publications have demonstrated its interest in the exploration of myelodysplastic disorders, multiparameter flow cytometry is perceived by many laboratory hematologists as dif...

Deferasirox (DFX) is an orally administered iron chelator approved for use in patients with transfusion-dependent iron overload due to myelodysplastic syndromes (MDS). The safety and efficacy of DFX h...

Medical and Biotech [MESH] Definitions

Myelodysplastic-myeloproliferative Diseases

Clonal myeloid disorders that possess both dysplastic and proliferative features but are not properly classified as either MYELODYSPLASTIC SYNDROMES or MYELOPROLIFERATIVE DISORDERS.

Hematopoietic Cell Growth Factors

These growth factors comprise a family of hematopoietic regulators with biological specificities defined by their ability to support proliferation and differentiation of blood cells of different lineages. ERYTHROPOIETIN and the COLONY-STIMULATING FACTORS belong to this family. Some of these factors have been studied and used in the treatment of chemotherapy-induced neutropenia, myelodysplastic syndromes, and bone marrow failure syndromes.

Myelodysplastic Syndromes

Clonal hematopoietic stem cell disorders characterized by dysplasia in one or more hematopoietic cell lineages. They predominantly affect patients over 60, are considered preleukemic conditions, and have high probability of transformation into ACUTE MYELOID LEUKEMIA.

Hematologic Neoplasms

Neoplasms located in the blood and blood-forming tissue (the bone marrow and lymphatic tissue). The commonest forms are the various types of LEUKEMIA, of LYMPHOMA, and of the progressive, life-threatening forms of the MYELODYSPLASTIC SYNDROMES.

Aortic Arch Syndromes

Conditions resulting from abnormalities in the arteries branching from the ASCENDING AORTA, the curved portion of the aorta. These syndromes are results of occlusion or abnormal blood flow to the head-neck or arm region leading to neurological defects and weakness in an arm. These syndromes are associated with vascular malformations; ATHEROSCLEROSIS; TRAUMA; and blood clots.

More From BioPortfolio on "Safety, Tolerability, and Efficacy of Deferasirox in MDS"